Wednesday, November 22, 2023

New CRISPR Treatment for Sickle Cell Disease

Sickle Cell Disease(SCD) is an autosomal recessive genetic abnormality affecting over 100,000 Americans. Instead of hemoglobin's regular disc shape, SCD causes hemoglobin cells to become sickle-shaped making them inefficient in carrying oxygen. This abnormality leads to a host of symptoms, such as extreme pain and fatigue, anemia, and an increased risk of stroke. Currently, the only treatment for this potentially debilitating disease is a bone marrow transplant. Still, even this is only a temporary fix, leaving the affected needing multiple transplants over the course of their life. A new treatment using CRISPR gene editing technology uses the patient's blood-forming stem cells and converts them into fetal Hemoglobin(HbF). The HbF then converts to regularly functioning hemoglobin cells, decreasing the amount of SCD episodes caused by the misshapen hemoglobin. Of the 30 participants in the Exa-cell study, 29 experienced no severe blood vessel blockage and minimal symptoms overall. All 30 study participants avoided hospitalization for 12 months after the treatment was performed. Though expected to be approved due to its high efficacy, the exa-cell treatment is still pending FDA approval. Though the long-term effects of this technique require further study, considering the significance of this CRISPR gene editing technique on people affected by SCD I feel it would be a step in the right direction for approval. I have always found the versatility of stem cells fascinating, whether they are used for cancer treatment, new drug research, or identifying genetic defects. I believe stem cells will play a pivotal role in future scientific breakthroughs.

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